Valoctocogene roxaparvovec-rvox (Intravenous)

Valoctocogene roxaparvovec-rvox injection is used to treat severe hemophilia A in patients who do not have antibodies to adeno-associated virus serotype 5 (AAV5).

This medicine should only be given by your doctor or someone under their supervision.

It is very important that your doctor checks you closely and at regular visits to make sure this medicine is working properly. Blood tests will be needed to check for unwanted effects.

Do not drink alcohol during treatment and for at least 1 year after receiving this medicine.

Women should not use this medicine, especially those who are pregnant or can become pregnant. This medicine can harm an unborn baby. It can also cause birth defects if the father is using it when his partner becomes pregnant. Males with partners who can become pregnant must use effective birth control during treatment and for at least 6 months after the last dose. If a pregnancy occurs while you are using this medicine, tell your doctor right away.

This medicine may cause infusion-related reactions. Check with your doctor right away if you have back pain, chest tightness, chills, fever, flushing, headache, nausea and vomiting, trouble breathing, or weakness.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you have blurred vision, chills, confusion, cough, diarrhea, dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position, fast, pounding, or irregular heartbeat or pulse, fever, hives, itching, skin rash, nausea, runny nose, shivering, sneezing, sweating, tingling of the throat, trouble breathing, unusual tiredness or weakness, or watery eyes after you receive the medicine.

Check with your doctor right away if you have clay-colored stool, dark urine, decreased appetite, fever, itching, loss of appetite, nausea and vomiting, skin rash, stomach pain, swelling of the feet or lower legs, unusual tiredness or weakness, or yellow eyes or skin. These may be symptoms of a serious liver problem.

This medicine may increase the risk for blood clots. Check with your doctor right away if you have pains in the chest, groin, or legs, especially in the calves of the legs, severe headaches that come on suddenly, sudden loss of coordination, sudden trouble breathing for no apparent reason, sudden slurred speech, or sudden vision changes.

While you are being treated with valoctocogene roxaparvovec-rvox injection, do not get any immunizations (vaccines) without your doctor's approval. Live virus vaccines should not be given with valoctocogene roxaparvovec-rvox.

Do not donate semen for at least 6 months after treatment.

Do not donate blood, organs, tissues, or cells for transplant after treatment.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

In deciding to use a medicine, you need to consider the risks of taking it against the benefits it may provide. This is a choice you will make with your doctor. For this medicine, keep the following in mind:

Allergies

Inform your doctor if you have ever had any unusual or allergic reactions to this medicine or any other medicines. Also let your healthcare provider know if you have any other allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Pediatric

There have not been appropriate studies on how age affects the use of valoctocogene roxaparvovec-rvox injection in children. Safety and effectiveness have not been established for this group.

Geriatric

Current studies have not shown any specific problems in older adults that would limit the usefulness of valoctocogene roxaparvovec-rvox injection in the elderly.

Breast Feeding

There are no adequate studies in women to determine the risk to infants when using this medication while breastfeeding. Consider the potential benefits and risks before taking this medication while breastfeeding.

Interactions with Medicines

Some medicines should not be taken together, while in other cases, two different medicines may be used together even if there is a possibility of an interaction. In these situations, your doctor may adjust the dose or take other precautions. When you are receiving this medicine, it is especially important for your healthcare provider to know if you are taking any of the following medicines. The following interactions have been chosen because of their potential significance and are not necessarily a complete list.

Using this medicine with any of the following medicines is generally not recommended, but may be necessary in some cases. If both medicines are prescribed, your doctor may change the dose or frequency of one or both of the medicines.

• Adenovirus Vaccine, Live
• Bacillus of Calmette and Guerin Vaccine, Live
• Cholera Vaccine, Live
• Dengue Tetravalent Vaccine, Live
• Ebola Zaire Vaccine, Live
• Efavirenz
• Influenza Virus Vaccine, Live
• Isotretinoin
• Measles Virus Vaccine, Live
• Mumps Virus Vaccine, Live
• Poliovirus Vaccine, Live
• Rotavirus Vaccine, Live
• Rubella Virus Vaccine, Live
• Smallpox Monkeypox Vaccine, Live Non-Replicating
• Smallpox Vaccine
• Typhoid Vaccine, Live
• Varicella Virus Vaccine, Live
• Yellow Fever Vaccine
• Zoster Vaccine, Live

Interactions with Food/Tobacco/Alcohol

Some medicines should not be used around the time of eating food or specific types of food due to possible interactions. Drinking alcohol or using tobacco with certain medicines may also lead to interactions. Talk to your healthcare provider about using your medicine with food, alcohol, or tobacco.

Other Medical Problems

Having other medical issues may affect how this medicine works. Make sure to tell your doctor if you have any other medical problems, especially:

• Allergy to mannitol or
• Active or uncontrolled infection or
• Liver disease (e.g., liver fibrosis or cirrhosis)—This medicine should not be used in patients with these conditions.

• Blood clots or
• Cancer or
• Heart or blood vessel disease or
• Infection or
• Liver disease (e.g., hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, and non-alcoholic steatohepatitis)—Use with caution. This may worsen these conditions.

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do, they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

• Back pain
• blurred vision
• chest tightness
• chills
• confusion
• cough
• diarrhea
• difficulty swallowing
• dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position
• fast heartbeat
• fever
• flushing
• headache
• hives, itching, skin rash
• nausea and vomiting
• puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
• runny nose
• shivering
• sweating
• tingling of the throat
• trouble breathing
• unusual tiredness or weakness
• watery eyes

Less common

• Loss of appetite
• stomach pain

Incidence not known

• Dark urine
• pains in the chest, groin, or legs, especially the calves of the legs
• severe headaches of sudden onset
• sudden loss of coordination
• sudden onset of slurred speech
• sudden vision changes
• yellow eyes or skin

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue, are bothersome, or if you have any questions about them:

More common

• General feeling of discomfort or illness
• headache
• unusual drowsiness, dullness, tiredness, weakness or feeling of sluggishness

Less common

• Stomach pain

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

In the U.S.

• Roctavian

Available Dosage Forms:

• Suspension

Therapeutic Class: Antihemophilic Agent

A doctor or other trained health professional will give you this medicine. It is given through a needle placed into one of your veins. It must be given slowly, so the needle will have to remain in place for at least 2 hours.